MOMENT registry: Patients with advanced non-small-cell lung cancer harboring exon 14 skipping treated with systemic therapy.

exon 14 ex14) skipping occurs in 3-4% of non-small-cell lung cancer (NSCLC) cases. Low frequency of this alteration necessitated open-label, single-arm trials to investigate MET inhibitors. Since broad MET biomarker testing was only recently introduced in many countries, there is a lack of historical real-world data from patients with ex14 skipping NSCLC receiving conventional therapies.

The Role of Value Analysis in Pressure Injury Prevention: A Quality Improvement Project.

Purpose: Global pressure injury (PI) statistics reveal that hospital-acquired pressure injuries (HAPIs) remain a substantial burden, with over 1 in 10 hospitalized adults being affected. The purpose of this analysis is to describe how consistent collection, analysis, and use of data allow hospitals to validate their clinical and economic outcomes and to adjust PI prevention strategies.

Participants And Setting: HAPI incidence data for acute care patients at a 280-bed regional community hospital in the Mid-Atlantic region of the United States (West Virginia) were collected from January 2012 to July 2023.

Safety and Efficacy of Deutetrabenazine at High versus Lower Daily Dosages in the ARC-HD Study to Treat Chorea in Huntington Disease.

Background: Huntington disease (HD) is a progressive neurodegenerative disease that causes psychiatric and neurological symptoms, including involuntary and irregular muscle movements (chorea). Chorea can disrupt activities of daily living, pose safety issues, and may lead to social withdrawal. The vesicular monoamine transporter 2 inhibitors tetrabenazine, deutetrabenazine, and valbenazine are approved treatments that can reduce chorea.

Mepolizumab real-world effectiveness in severe asthma with an eosinophilic phenotype and overlapping severe allergic asthma.

Background: Some patients with severe asthma have overlapping allergic and eosinophilic phenotypes and may be eligible for anti-eosinophilic or anti-IgE biologics.

Objective: This post hoc sub-analysis assessed real-world mepolizumab effectiveness in patients with overlapping allergic and eosinophilic phenotypes, using 1-year data from the international, prospective REALITI-A study.

Methods: The clinically significant asthma exacerbation (CSE) rate was assessed 1 year prior to (pre-treatment) and following (follow-up) mepolizumab treatment, stratified by baseline total IgE levels (tIgE; <60, 60-<190, 190-<550, and ≥550 kU/L), atopic status (yes/no/unknown), prior omalizumab use (yes/no), geographic baseline omalizumab eligibility (eligible/non-eligible), and baseline tIgE level and blood eosinophil count (BEC) threshold combinations (<81 or ≥81 kU/L and <300 or ≥300 cells/µL).

Point of view: Challenges in implementation of new immunotherapies for Alzheimer's disease.

The advancement of disease-modifying treatments (DMTs) for Alzheimer's disease (AD), along with the approval of three amyloid-targeting therapies in the US and several other countries, represents a significant development in the treatment landscape, offering new hope for addressing this once untreatable chronic progressive disease. However, significant challenges persist that could impede the successful integration of this class of drugs into clinical practice. These challenges include determining patient eligibility, appropriate use of diagnostic tools and genetic testing in patient care pathways, effective detection and monitoring of side effects, and improving the healthcare system's readiness by engaging both primary care and dementia specialists.

Determination of the Bioavailability of 3 Intranasal Formulations of Azelastine Hydrochloride in Healthy Male Volunteers.

The primary objective of the study was to determine the bioavailability of 2 new formulations of azelastine (AZE) hydrochloride (0.10% and 0.15% AZE) containing sorbitol and sucralose compared with the commercially available 0.

Evaluating the impact of changing inhaler color on perception of symptoms and disease burden in patients with asthma: the FEEL study.

Objectives: Patient perception of treatment effectiveness is key to optimizing adherence. This is potentially impacted by color, yet no such studies have been conducted in asthma. This study assessed the influence of pink vs.

On the correction factors for small field dosimetry in 1.5T MR-linacs.

. Clinical dosimetry in the presence of a 1.5 T magnetic field is challenging, let alone in case small fields are involved.

Clinical outcomes by baseline metastases in patients with renal cell carcinoma treated with lenvatinib plus pembrolizumab versus sunitinib: Post hoc analysis of the CLEAR trial.

Lenvatinib plus pembrolizumab significantly improved efficacy versus sunitinib in treatment of advanced renal cell carcinoma (aRCC) in the phase 3 CLEAR study. We report results of an exploratory post hoc analysis of tumor response data based on baseline metastatic characteristics of patients who received lenvatinib plus pembrolizumab versus sunitinib, at the final overall survival analysis time point of CLEAR (cutoff: July 31, 2022). Treatment-naïve adults with aRCC were randomized to: lenvatinib (20 mg PO QD in 21-day cycles) plus pembrolizumab (n = 355; 200 mg IV Q3W); lenvatinib plus everolimus (not reported here); or sunitinib (n = 357; 50 mg PO QD; 4 weeks on/2 weeks off).

A phase I, randomized, placebo-controlled trial to evaluate the pharmacokinetics, safety, and tolerability of nirsevimab in healthy Chinese adults.

Respiratory syncytial virus (RSV) is the leading cause of lower respiratory tract infection (LRTI) in infants worldwide. Nirsevimab, an extended half-life monoclonal antibody against RSV, is approved in China for the prevention of RSV lower respiratory tract disease in infants; however, global nirsevimab trials did not enroll Chinese infants. To inform the investigation of nirsevimab for the prevention of RSV LRTI in Chinese infants, this Phase I, randomized, placebo-controlled trial evaluated the pharmacokinetics (PK) and safety of nirsevimab in healthy Chinese adults.

Effects of Pemafibrate on LDL-C and Related Lipid Markers in Patients with MASLD: A Sub-Analysis of the PEMA-FL Study.

Aim: In the PEMA-FL study in patients with metabolic dysfunction-associated steatotic liver disease (MASLD), pemafibrate was shown to significantly decrease low-density lipoprotein cholesterol (LDL-C) levels. We aimed to investigate the mechanisms of pemafibrate-induced LDL-C reduction in patients with MASLD by conducting an additional sub-analysis of the PEMA-FL study.

Methods: The PEMA-FL study randomized 118 patients with MASLD to receive pemafibrate or placebo for 72 weeks.

Development of Real-Time Surveillance for Serious Adverse Events in a Pragmatic Clinical Trial Using National Registers in Finland.

Purpose: We developed a hybrid safety surveillance approach for a large, pragmatic clinical trial of a high-dose quadrivalent influenza vaccine (QIV-HD), using both active and passive data collection methods. Here, we present the methods and results for the passive register-based surveillance of serious adverse events (SAEs), which replaced conventional SAE reporting during the trial.

Patients And Methods: The trial recruited over 33,000 older adults of whom 50% received the QIV-HD while the rest received a standard-dose vaccine (QIV-SD) as a control vaccine.

Advancements in Ocular Modelling and Simulations: Key Considerations and Case Studies.

This review paper discusses the key aspects of ocular biopharmaceutics, with emphasis on the crucial role played by ocular compartmental modelling and simulation in deciphering physiological conditions related to various eye diseases. It describes eye's intricate structure and function and the need for precise and targeted drug delivery systems to address prevalent eye conditions. The review categorizes and discusses various formulations employed in ocular drug delivery, delineating their respective advantages and limitations.

A Principled Approach to Adjust for Unmeasured Time-Stable Confounding of Supervised Treatment.

We propose a novel method to adjust for unmeasured time-stable confounding when the time between consecutive treatment administrations is fixed. We achieve this by focusing on a new-user cohort. Furthermore, we envisage that all time-stable confounding goes through the potential time on treatment as dictated by the disease condition at the initiation of treatment.

Current State and New Horizons in Applications of Physiologically Based Biopharmaceutics Modeling (PBBM): A Workshop Report.

This report summarizes the proceedings for Day 3 of the workshop titled "". This day focused on the current and future drug product quality applications of PBBM from the innovator and generic industries as well as the regulatory agencies perspectives. The presentations, which included several case studies, covered the applications of PBBM in generic drug product development, applications of virtual bioequivalence trials to support formulation bridging and the utility of absorption modeling in clinical pharmacology assessments.

Quadrivalent meningococcal tetanus toxoid-conjugate booster vaccination in children aged 10-12 years: phase III randomized trial complementary analysis of immune persistence 3-6 years after priming.

Immune persistence following primary vaccination with a single dose of meningococcal quadrivalent conjugate vaccines, MenACYW-TT/MCV4-CRM, at age 10-12 years was demonstrated. Most participants primed with MenACYW-TT and MCV4-CRM maintained seroprotective titers against all serogroups, suggesting continued protection. Priming with MenACYW-TT resulted in higher persistent titers for serogroups C, W, and Y than MCV4-CRM.

Incidence and prevalence of antiphospholipid syndrome (APS) in the USA (2016-2019): a retrospective database study.

Objective: Few epidemiological studies are reported in the published literature on the incidence or prevalence of antiphospholipid syndrome (APS), and available results are heterogeneous. This study aimed to estimate the incidence and prevalence of APS in the USA, overall and by APS subtype.

Design: A retrospective analysis of APS disease incidence and a cross-sectional analysis of disease prevalence.

Screening and diagnosis of pulmonary hypertension associated with chronic lung disease (PH-CLD): A consensus statement from the pulmonary vascular research institute's innovative drug development initiative-group 3 pulmonary hypertension.

Pulmonary hypertension (PH) is a frequent complication of chronic lung disease (CLD). However, PH is difficult to diagnose early since accompanying symptoms overlap and are similar to those of the underlying CLD. In most cases the PH is mild to moderate and therefore physical signs may be absent or subtle.

Double-Blind, Placebo-Controlled Trial of the Efficacy and Safety of Azelastine Hydrochloride in Children with Perennial Allergic Rhinitis.

Introduction: Allergic rhinitis (AR) affects up to 40% of the pediatric population. The US practice parameter recommends the use of intranasal antihistamines (INAH) or INCS as first-line therapy for the treatment of AR. Although not directly targeted to children, the recent US practice parameters proposed INAH as first-line therapy whereas the ARIA guidelines did not.

Physiologically Based Biopharmaceutics Modeling Coupled with Biopredictive Dissolution in Development of Bioequivalent Formulation for Mesalamine Enteric Coated Tablet: A Tough Nut to Crack.

Mesalamine is a locally acting anti-inflammatory drug used to treat mild to moderate ulcerative colitis. Because of complex formulation principle and high in vivo variability, development of bioequivalent formulation for mesalamine is challenging. Further, fed state possess significant challenges for bioequivalence (BE) due to interplay of multiple factors.

An Open-Label, Phase III Study to Assess the Efficacy and Safety of Cysteamine Ophthalmic Solution 0.55% in Japanese Cystinosis Patients.

Purpose: Corneal cystine accumulation results in photophobia and affects patients' quality of life. We assessed the efficacy and safety of cysteamine 0.55% solution in Japanese cystinosis patients with corneal cystine crystals for 52 weeks.